ESPE Abstracts

Context: Off-label and unlicensed use is widespread in medical therapy of children. Since 2007 there is an EU regulation, which includes the need for a pediatric investigation plan (PIP) in the drug approval process. In 2018, hydrocortisone granules in capsules for opening (Alkindi®) became the first medicine to be approved for the treatment of adrenal insufficiency in children from birth.

Methods: An anonymised online survey (SurveyMonkey) was created to collect information on the underlying diagnosis, frequency and compliance with glucocorticoid therapy, use of glucocorticoids (stress dosing) during illness, and acceptance of or satisfaction with the hydrocortisone granules in capsules for opening. Participants were recruited by informing them about the survey during their visits in the outpatient clinic and with the help of self-help organisations.

Results: The following are preliminary results of the ongoing survey of parents of children aged 0-18 years. In 92% of those who have participated so far, children are treated by a paediatric endocrinologist. The children of most participants where suffering from CAH (67%). All children were treated with hydrocortisone as glucocorticoid therapy. In case of stress dosing, the usual medication was increased (90%), a prednisolone suppository was additionally administered (50%), licensed hydrocortisone tablets are used (40%) or an emergency injection of hydrocortisone is given (10%). In summary, 92% of the participants were satisfied with the new hydrocortisone preparation. For parents, the simplicity of handling (100%), the accuracy of dosing (70%) and an existing marketing authorisation (60%), but less the taste (30%) are important in the treatment of their children.

Conclusion: The hydrocortisone granules in capsules for opening show a good acceptance among parents. For relatives, important qualities of drug therapy in childhood are simplicity of handling especially for the medication of young children.